Moderately severe hemophilia B is a form of hemophilia B characterized by factor IX deficiency leading to abnormal bleeding as a result of minor injuries, or following surgery or tooth extraction.
Comprehensive, easy-to-understand information about this condition
How we create this content →The limited documentation surrounding moderately severe hemophilia B is largely due to its rarity, affecting fewer than 10 individuals per million. This extreme rarity restricts the scope of systematic clinical studies and comprehensive phenotypic characterization. As a result, many aspects of the condition, including its clinical features and genetic underpinnings, remain poorly understood.
To navigate your condition effectively, consider consulting a hematologist with specific expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition closely. Additionally, you may explore clinical trials that could offer access to emerging therapies. Resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information and support. While no specific patient organizations are identified, connecting with broader hemophilia communities may also be beneficial.
There are several orphan drugs designated for moderately severe hemophilia B, including Coagulation Factor IX (recombinant), concizumab-mtci, etranacogene dezaparvovec-drlb, fidanacogene elaparvovec-dzkt, and fitusiran. Additionally, there are 9 active clinical trials exploring various treatment options. You can find more information about these trials at ClinicalTrials.gov [here](https://clinicaltrials.gov/search?cond=moderately%20severe%20hemophilia%20B). This research offers hope for future treatment advancements.
Actionable guidance for navigating care for moderately severe hemophilia B
To navigate your condition effectively, consider consulting a hematologist with specific expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition closely. Additionally, you may explore clinical trials that could offer access to emerging therapies. Resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information and support. While no specific patient organizations are identified, connecting with broader hemophilia communities may also be beneficial.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
The limited documentation surrounding moderately severe hemophilia B is largely due to its rarity, affecting fewer than 10 individuals per million. This extreme rarity restricts the scope of systematic clinical studies and comprehensive phenotypic characterization. As a result, many aspects of the condition, including its clinical features and genetic underpinnings, remain poorly understood.
To navigate your condition effectively, consider consulting a hematologist with specific expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition closely. Additionally, you may explore clinical trials that could offer access to emerging therapies. Resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information and support. While no specific patient organizations are identified, connecting with broader hemophilia communities may also be beneficial.
There are several orphan drugs designated for moderately severe hemophilia B, including Coagulation Factor IX (recombinant), concizumab-mtci, etranacogene dezaparvovec-drlb, fidanacogene elaparvovec-dzkt, and fitusiran. Additionally, there are 9 active clinical trials exploring various treatment options. You can find more information about these trials at ClinicalTrials.gov [here](https://clinicaltrials.gov/search?cond=moderately%20severe%20hemophilia%20B). This research offers hope for future treatment advancements.
Actionable guidance for navigating care for moderately severe hemophilia B
To navigate your condition effectively, consider consulting a hematologist with specific expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition closely. Additionally, you may explore clinical trials that could offer access to emerging therapies. Resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information and support. While no specific patient organizations are identified, connecting with broader hemophilia communities may also be beneficial.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
The limited documentation surrounding moderately severe hemophilia B is largely due to its rarity, affecting fewer than 10 individuals per million. This extreme rarity restricts the scope of systematic clinical studies and comprehensive phenotypic characterization. As a result, many aspects of the condition, including its clinical features and genetic underpinnings, remain poorly understood.
To navigate your condition effectively, consider consulting a hematologist with specific expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition closely. Additionally, you may explore clinical trials that could offer access to emerging therapies. Resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information and support. While no specific patient organizations are identified, connecting with broader hemophilia communities may also be beneficial.
There are several orphan drugs designated for moderately severe hemophilia B, including Coagulation Factor IX (recombinant), concizumab-mtci, etranacogene dezaparvovec-drlb, fidanacogene elaparvovec-dzkt, and fitusiran. Additionally, there are 9 active clinical trials exploring various treatment options. You can find more information about these trials at ClinicalTrials.gov [here](https://clinicaltrials.gov/search?cond=moderately%20severe%20hemophilia%20B). This research offers hope for future treatment advancements.
Actionable guidance for navigating care for moderately severe hemophilia B
To navigate your condition effectively, consider consulting a hematologist with specific expertise in bleeding disorders, particularly hemophilia. They can provide tailored management strategies and monitor your condition closely. Additionally, you may explore clinical trials that could offer access to emerging therapies. Resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information and support. While no specific patient organizations are identified, connecting with broader hemophilia communities may also be beneficial.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
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